ArticleScientific reports2015
Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCs.
Article in Scientific reports, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 96 papers.
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96 citing papers in PubMed, 147 citations in OpenAlex.
- Precision base-editing of the cryptic 3' acceptor site to correct the RNA splicing defect of βMolecular biomedicine · 2026Article
- CRISPR Technology: Transforming the Future of Medicine and Diagnostics.Biochemistry · 2025Review
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- Genetic Engineering in Hematopoietic Stem Cells for β-Hemoglobinopathies Treatment: Advances, Challenges, and Clinical Translation.International journal of hematology-oncology and stem cell research · 2025Review
- CRISPR/Cas-edited iPSCs and mesenchymal stem cells: a concise review of their potential in thalassemia therapy.Frontiers in cell and developmental biology · 2025Review
- Gene Editing of the Endogenous Cryptic 3' Splice Site Corrects the RNA Splicing Defect in the βHuman gene therapy · 2024Article
- Development of pathophysiologically relevant models of sickle cell disease and β-thalassemia for therapeutic studies.Nature communications · 2024Article
- Advances and challenges in gene therapy strategies for pediatric cancer: a comprehensive update.Frontiers in molecular biosciences · 2024Review
- CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia.Human genetics · 2023Review
- CRISPR/Cas9 Ablated BCL11A Unveils the Genes with Possible Role of Globin Switching.Advanced pharmaceutical bulletin · 2023Article
- Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells.Stem cell reviews and reports · 2023Review
- Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies.International journal of molecular sciences · 2023Review
- Immortalized erythroid cells as a novel frontier for in vitro blood production: current approaches and potential clinical application.Stem cell research & therapy · 2023Review
- CRISPR/Cas9 andInternational journal of molecular sciences · 2023Article
- High-Efficiency CRISPR/Cas9-Mediated Correction of a Homozygous Mutation in Achromatopsia-Patient-Derived iPSCs.International journal of molecular sciences · 2023Article
- Combined approaches for increasing fetal hemoglobin (HbF) andFrontiers in genome editing · 2023Review
- Advances in CRISPR therapeutics.Nature reviews. Nephrology · 2023Review
- Review
- Genetic correction of haemoglobin E in an immortalised haemoglobin E/beta-thalassaemia cell line using the CRISPR/Cas9 system.Scientific reports · 2022Article
- Gene Editing-Based Technologies forBiology · 2022Review
36 more citing papers are in PubMed but not listed here.
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9 authors at 1 institution in 1 country.
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Abstract
β-Thalassemia is one of the most common genetic blood diseases and is caused by either point mutations or deletions in the β-globin (HBB) gene. The generation of patient-specific induced pluripotent stem cells (iPSCs) and subsequent correction of the disease-causing mutations may be a potential therapeutic strategy for this disease. Due to the low efficiency of typical homologous recombination, endonucleases, including TALENs and CRISPR/Cas9, have been widely used to enhance the gene correction efficiency in patient-derived iPSCs. Here, we designed TALENs and CRISPR/Cas9 to directly target the intron2 mutation site IVS2-654 in the globin gene. We observed different frequencies of double-strand breaks (DSBs) at IVS2-654 loci using TALENs and CRISPR/Cas9, and TALENs mediated a higher homologous gene targeting efficiency compared to CRISPR/Cas9 when combined with the piggyBac transposon donor. In addition, more obvious off-target events were observed for CRISPR/Cas9 compared to TALENs. Finally, TALENs-corrected iPSC clones were selected for erythroblast differentiation using the OP9 co-culture system and detected relatively higher transcription of HBB than the uncorrected cells. This comparison of using TALENs or CRISPR/Cas9 to correct specific HBB mutations in patient-derived iPSCs will guide future applications of TALENs- or CRISPR/Cas9-based gene therapies in monogenic diseases.
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